Neurological implications of chimeric antigen receptor-T cell therapy

Raffoul, DG; Roos, I; Mulazzani, M; Anderson, MA; Monif, M; Garber, J; van der Walt, A; Massey, J; Kuznetsova, V; Nesbitt, C; Dowling, M; Ruderman, I; Lasocki, A; Tam, CS; Harrison, SJ; Kalincik, T

Author(s): Raffoul, DG; Roos, I; Mulazzani, M; Anderson, MA; Monif, M; Garber, J; van der Walt, A; Massey, J; Kuznetsova, V; Nesbitt, C; Dowling, M; Ruderman, I; Lasocki, A; Tam, CS; Harrison, SJ; Kalincik, T;
Details: Publication Year 2026,Volume 8,Issue #1,Page e001491
Journal Title: BMJ Neurology Open
Publication Type: Review
Abstract: Chimeric antigen receptor (CAR)-T cell therapies have transformed the management of relapsed or refractory haematological malignancies and are now being adapted for severe B-cell mediated autoimmune disease, including neuroimmunological disorders. Their appeal lies in deep and durable B-cell depletion within lymphoid tissues and target organs, with the potential to induce long-lasting, treatment-free remission. At the same time, broader application is constrained by serious toxicities, in particular, neurological complications such as immune effector cell-associated neurotoxicity syndrome (ICANS) and more recently recognised movement, cerebellar, cranial nerve and peripheral nerve syndromes. In this review, we outline the biological principles of CAR-T cell therapy and summarise the emerging experience in neuroimmunology. We draw on data from both oncology and early autoimmune trials to describe the clinical spectrum, timing and proposed mechanisms of CAR-T cell-related neurotoxicity and to distinguish ICANS from later and more focal toxicities. As CAR-T cell therapies move from cancer centres into the care of patients with refractory neuroimmunological disease, neurologists will need a detailed understanding of both their therapeutic promise and their neurological risks, and close collaboration within multidisciplinary teams will be essential to deliver these treatments safely.
Publisher: BMJ
Keywords: Haematology; Multiple sclerosis; Myasthenia; Neuroimmunology
Department(s): Internal Medicine; Haematology; Cancer Imaging
Publisher's Version: https://doi.org/10.1136/bmjno-2025-001491
Open Access at Publisher's Site: https://doi.org/10.1136/bmjno-2025-001491
Terms of Use/Rights Notice: Refer to copyright notice on published article.

Creation Date: 2026-04-07 03:20:35

Last Modified: 2026-04-07 03:20:52